A First-in-Human Study in Pediatric Patients With Ocular CLN2 Disease

Inclusion Criteria: A participant is eligible to be included in the study only if all of the following criteria apply: Has biallelic CLN2 mutations. Has decreased leukocyte TPP1 activity. Has clinical signs or symptoms consistent with CLN2 disease (eg, developmental delay, developmental decline, seizure, vision loss, or other signs/symptoms) OR an older sibling with confirmed CLN2 diagnosis. Is currently receiving biweekly ICV ERT treatment with cerliponase alfa. Meets baseline disease condition according to age, retinal thickness, and visual acuity criteria ( varies by treatment arm) Exclusion Criteria: Participants are excluded from the study if any of the following criteria apply: Any ocular or systemic condition that, in the opinion of the investigator, would prevent administration and evaluation of the investigational product or interpretation of participant safety or study results (eg, significant lens or corneal opacities, glaucoma, amblyopia, gross retinal anatomical abnormality, etc). Prior participation in a gene therapy study Prior participation in another ocular clinical trial, except an intravitreal cerliponase alfa trial where a subject has received a maximum of 3 injections Prior intraocular injections of any kind, except an intravitreal cerliponase alfa trial where a subject has received a maximum of 3 injections Participation in a clinical study with an investigational drug in the past six months prior to screening, except for intracerebroventricular cerliponase alfa. Ocular surgery within the prior six months. Known sensitivity or contraindications to medications planned for use in the peri-operative period. Contraindications to systemic immunosuppression Any other condition that would not allow the potential participant to complete follow-up examinations during the study or, in the opinion of the investigator, makes the potential participant unsuitable for the study